Huntington disease and related disorders

This clinical trial tests an oral drug called SKY-0515 in 400 adults with Huntington's disease. The goal is to see if it can lower harmful proteins linked to the disease and improve symptoms like movement and thinking. Participants take the drug or a placebo daily for 18 months.

This study tests whether the drug pridopidine can slow the worsening of Huntington's disease (HD) in adults. About 400 participants will take either pridopidine or a placebo twice daily for one year, then everyone can receive pridopidine for two more years. The goal is to see if …

This early-stage study tests an experimental drug called ALN-HTT02 in 66 adults with Huntington's disease. The drug is given as a single injection into the spine to see if it is safe and how it affects the body. The goal is to reduce levels of the harmful protein that causes the …

This study tests a new drug called SRP-1005 in 32 adults with Huntington's disease. It is the first time this drug is being tested in humans, so the main goal is to check safety and how the body processes the drug. Participants will receive either SRP-1005 or a placebo by injecti…

This Phase 3 trial tests whether the drug votoplam can slow the progression of Huntington's disease in people with early symptoms. About 770 adults aged 21 to 70 with a confirmed genetic diagnosis will receive either votoplam or a placebo. The main goal is to see if votoplam pres…

This first-in-human trial is testing an experimental drug called VO659 in people with spinocerebellar ataxia type 1, type 3, or Huntington's disease. The drug is designed to target the genetic repeats that cause these conditions, potentially slowing their progression. The study a…

This study tests a new gene therapy called SPK-10001 for people with Huntington's disease. It involves a one-time infusion into specific areas of the brain. The main goal is to see if it is safe and if it can slow down the worsening of movement and daily function. About 53 adults…

This early-phase trial tests whether implanting human neural stem cells into the brain is safe for people with Huntington's disease. Twenty-one adults with confirmed Huntington's will receive the stem cell therapy and be monitored for side effects over 12 weeks and up to a year. …

This study is testing a new blood test that can diagnose single-gene disorders in unborn babies using a sample from the mother. The test looks at fetal DNA found in the mother's blood, which is safer than traditional invasive methods that carry a small risk of miscarriage. Resear…

This early-stage study tests a new radioactive tracer called [11C]CHDI-00491009 to see if it can safely and accurately detect harmful protein clumps (mutant huntingtin) in the brains of people with Huntington's disease. The study involves 27 participants, including healthy volunt…

This early-stage trial tests whether low-intensity focused ultrasound (LIFU) can safely improve motor symptoms in 24 adults with Huntington's disease. Participants receive ultrasound pulses to a brain region involved in movement control. The study focuses on safety and finding th…

This study compares two types of exercise-video game (exergame) programs for people with Huntington's disease. One program mixes physical and mental exercises together, while the other does them separately. Researchers want to see which approach improves balance, thinking, and da…

This study is testing whether a gentle brain stimulation device (tDCS) used at home is safe and acceptable for people with early-to-moderate Huntington's disease. Ten participants will use the device daily for several weeks while researchers track side effects, mood, thinking ski…

This pilot study tests whether a gentle brain stimulation device (tDCS) used at home can help with cognitive and behavioral symptoms in Huntington's disease. Sixteen adults with early to moderate Huntington's will use the device daily for 30 minutes, alternating between real and …

This pilot study is testing whether adding music therapy to standard care can reduce irritability and impulsivity in 15 people with early-stage Huntington's disease. Participants will use a digital music therapy tool over 3 months. The goal is to find a safe, non-drug way to mana…

This study tests whether a computer-based training program can help people with Huntington's disease better understand others' emotions and social cues. Twenty participants will use the program at home and come in for follow-up visits. The goal is to see if this approach is pract…

This study uses special PET scans to measure inflammation in the brains of people with Alzheimer's, Parkinson's, ALS, and other neurodegenerative diseases. Researchers will compare these scans to those from healthy volunteers to see if inflammation levels differ. The goal is to d…

This study screens 360 adults with neurodegenerative disorders like ALS, frontotemporal dementia, and progressive supranuclear palsy. Researchers will use medical history, physical exams, memory tests, movement analysis, MRI scans, and other tests to find common features and diff…

This study aims to create a network of biobanks across Italy that collect and store biological samples and health data from people with rare diseases and their families. The goal is to standardize how samples are collected and shared, so researchers can access high-quality materi…

This study is testing whether wearable devices like smartwatches can accurately track movement problems in people with Huntington's disease. About 100 participants, including those with Huntington's and healthy volunteers, will wear sensors and complete surveys and interviews at …

This study uses a special PET scan tracer, [18F]MH.MZ, to measure serotonin 2A receptors in the brains of people with neurodegenerative diseases like Parkinson's. Researchers want to see if these receptors differ between patients with psychosis and healthy people of the same age.…

This study follows people with several types of spinocerebellar ataxia (SCA) — rare genetic diseases that cause balance, coordination, and speech problems — to learn how these conditions change over time. Researchers will collect blood samples, perform neurological exams, and use…

This study aims to collect cerebrospinal fluid and blood samples from 2,500 people at various stages of Huntington's disease, as well as healthy volunteers. The goal is to find biological markers that can help researchers develop new treatments. Participants must be between 11 an…

This study creates a worldwide database for people with DRPLA, a rare genetic brain disorder. Participants or their caregivers fill out questionnaires about symptoms, daily life, and disease impact. The goal is to gather information to help researchers better understand DRPLA and…

This study looks at how Huntington disease affects social skills, like understanding others' feelings and intentions. Researchers will test a new tool called DMASC-MC in 40 adults with early Huntington disease and compare results to healthy volunteers. The goal is to find better …

This study looks at two thinking and memory tests—the Coding Test and the SAGE test—to see if they can help doctors better understand and track Huntington's disease. Researchers will compare these tests with standard ones used in clinics. The goal is to find easier or better ways…

This study looks at a protein called BDNF, which helps keep brain cells healthy, in people with Huntington's disease. Researchers will measure BDNF and related markers in spinal fluid and blood, and compare them to healthy controls. The goal is to find biological signs that could…

This study uses non-invasive eye scans (OCT and OCTA) to look for changes in the retina that might be linked to brain diseases like Alzheimer's, Parkinson's, and multiple sclerosis. Researchers aim to find early markers that could help diagnose or track these conditions. The stud…

This study looks at whether a test called the Hinting Task can pick up on social thinking difficulties in people with Huntington's disease. Social thinking helps us understand hints and other people's perspectives, and problems with it can affect daily life. Researchers will comp…

This study looks at how spinocerebellar ataxia (SCA) affects the eyes and vision. Researchers will examine 60 adults with SCA types 1, 2, 3, or 27B, including those with early symptoms and those who are not yet symptomatic. The goal is to measure vision changes and eye damage usi…

This study aims to collect health information from 600 adults with Huntington disease or those who carry the gene but have no symptoms yet. Participants will answer online questions from home over 24 months. The goal is to learn how the disease progresses and what factors might a…